How we deliver therapeutics.

Our LNP Platform

Our industry-leading lipid nanoparticle (LNP) platform has enabled clinical development of the most advanced RNA-based therapeutics.

Our LNP platform is the only clinically-validated LNP delivery technology with safety and efficacy evaluated in over 400 patients across multiple clinical programs. It is the first and only LNP platform to enable an approved therapy and has enabled the first siRNA NDA submission filing through its licensed use by Alnylam Pharmaceuticals for the delivery of patisiran, a therapy to treat patients with hereditary ATTR amyloidosis.

We intend to use our leadership position in LNP and the strength of the platform’s intellectual property to develop our own LNP products and to pursue multiple partnership opportunities.

Our Ligand Conjugate Platform

Our proprietary ligand conjugate delivery platform has been designed for RNAi therapeutics targeting the liver.

We intend to develop numerous RNAi-conjugate products in-house targeting a wide range of genetically-defined diseases.